Ignite Creation Date:
2025-12-26 @ 2:40 PM
Ignite Modification Date:
2026-01-01 @ 5:52 AM
Study NCT ID:
NCT04925206
Status:
TERMINATED
Last Update Posted:
2025-03-30
First Post:
2021-06-04
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia
Sponsor:
EdiGene (GuangZhou) Inc.
Organization:
Study Overview
Official Title:
A Multicenter, Open Label Phase 1 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects with Transfusion Dependent Β-Thalassaemia
Status:
TERMINATED
Status Verified Date:
2025-03
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Development plan change
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
ET-01
Brief Summary:
This is a single-arm, open label, multi-center, single-dose phase 1 study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using ET-01.
Detailed Description:
None
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?: